FDA Approves Ocrelizumab for Pediatric Patients With Relapsing-Remitting Multiple Sclerosis

The FDA has approved intravenous ocrelizumab (Ocrevus; Genentech) for the treatment of relapsing-remitting multiple sclerosis (RRMS) in pediatric patients aged 10 years and older who weigh at least 55 pounds, expanding a high-efficacy anti-CD20 therapy into a younger population with often highly active disease.¹

With the decision, ocrelizumab becomes an FDA-approved treatment option for pediatric RRMS, a group that accounts for an estimated 3% to 5% of MS cases globally and approximately 5000 to 10,000 children and adolescents in the United States. The approval was supported by data from the phase 3 OPERETTA 2 study (NCT05123703), which compared ocrelizumab with fingolimod (Gilenya; Novartis), previously the only FDA-approved therapy for pediatric RRMS.

“This approval represents a landmark for children living with MS in the US and their families, which can help close the longstanding gap in high-efficacy treatment options for children aged 10 and older,” Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development at Genentech, said in a statement.¹ “By bringing a decade of efficacy and safety data to this younger population, Ocrevus may reduce relapses and potentially redefine what’s possible for their future.”

OPERETTA 2 was a randomized, double-blind, double-dummy, multicenter trial in which 187 pediatric patients were assigned to either ocrelizumab 600 mg intravenously every 24 weeks or fingolimod 0.5 mg orally once daily. The study met its primary objective, with ocrelizumab demonstrating noninferiority to fingolimod in reducing annualized relapse rate (ARR). Patients treated with ocrelizumab had a 48% relative reduction in ARR compared with fingolimod (rate ratio, 0.52; 95% CI, 0.19-1.33).^1,2

MRI outcomes also favored ocrelizumab. Treatment was associated with 48% fewer new or enlarging T2 lesions compared with fingolimod (rate ratio, 0.52; 95% CI, 0.36-0.76; P = .001) and 87% fewer gadolinium-enhancing T1 lesions at 12 weeks (rate ratio, 0.13; 95% CI, 0.03-0.41; P = .001).^1,2 The trial cohort had a median baseline age of 15 years, a median Expanded Disability Status Scale score of 1.5, and substantial baseline MRI disease burden, including a mean of 57 T2 lesions and at least 1 gadolinium-enhancing lesion in 47.1% of participants.²

The data were previously presented at the 2025 ECTRIMS Congress, at which time Brenda Banwell, MD, pediatrician-in-chief and codirector at Johns Hopkins Children’s Center, and colleagues noted that pediatric-onset MS is frequently characterized by high inflammatory activity and that the therapeutic goal is to suppress both clinical and MRI disease activity while preserving safety and tolerability. “More [disease-modifying therapies] options are required for POMS,” they wrote.² “If approved, ocrelizumab has the potential to be a highly effective and well tolerated treatment for POMS, with an already well-established safety profile in adults.”

The safety profile of ocrelizumab in OPERETTA 2 was consistent with prior adult experience. Serious adverse events and serious infections were infrequent and balanced across groups; no adverse events led to treatment withdrawal among patients receiving ocrelizumab, compared with 3 withdrawals in the fingolimod arm.¹ Infusion-related reactions and respiratory tract infections were among the primary adverse events in the ocrelizumab group, with most reactions mild or moderate and all resolving.²

Ocrelizumab is a recombinant humanized monoclonal antibody directed against CD20-expressing B cells, a cell population implicated in inflammatory demyelination and axonal injury in MS.³ The therapy was initially approved by the FDA in 2017 for adults with relapsing forms of MS and primary progressive MS, becoming the first approved therapy for PPMS at that time.⁴ Since then, it has become a foundational high-efficacy option in adult MS care, with intravenous dosing typically administered every 6 months after initial split dosing.

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The pediatric approval also builds on prior dose-selection and safety work from the phase 1 OPERETTA 1 study (NCT04075266), which evaluated ocrelizumab in pediatric patients with RRMS and informed the dose used in the phase 3 trial.² In that earlier study, ocrelizumab prevented relapses through 96 weeks and was generally well tolerated, providing a bridge between adult data and the confirmatory pediatric study.²

This decision adds to the life cycle of development for ocrelizumab. In 2024, the FDA approved a subcutaneous formulation, Ocrevus Zunovo, for adults with relapsing forms of MS and PPMS, offering a shorter administration option compared with IV infusion. The approval was based on the phase 3 OCARINA 2 study, which showed noninferior exposure with subcutaneous ocrelizumab 920 mg compared with IV ocrelizumab 600 mg, while maintaining similar disease activity control through 24 weeks.⁵

For pediatric MS care, the new approval may be particularly meaningful given the long-term implications of early disease activity. Children and adolescents with RRMS often experience frequent relapses and high MRI lesion accrual early in the disease course, raising concern for cumulative disability and cognitive effects over time. By expanding ocrelizumab into patients as young as 10 years, clinicians now have an additional high-efficacy option aimed at early disease suppression.

“Growing up with MS, I know the frustration of being dismissed and the fear of what comes next,” Emily Blosberg, founder of Mr. Oscar Monkey, who was diagnosed with MS at age 15, said in a statement.¹ “Having an FDA-approved, high-efficacy treatment option like Ocrevus available for age-appropriate children and adolescents is a game changer. It means the next generation of patients won't have to wait for answers—they have an opportunity to take control of their disease early and potentially stop relapses and brain lesions before they have a chance to take a toll.”

REFERENCES
1. Genentech. FDA approves Ocrevus (ocrelizumab) for pediatric relapsing multiple sclerosis. News release. Published May 2026. Accessed May 2026. https://www.gene.com/media/press-releases
2. Banwell B, Kotulska-Jozwiak K, Rostásy K, et al. Efficacy and safety of ocrelizumab compared with fingolimod in paediatric relapsing-remitting MS: results of the phase III OPERETTA 2 study. Presented at: ECTRIMS Congress; September 24-26, 2025; Barcelona, Spain. Late-Breaking Abstract O130. https://ectrims.eu
3. Schreiner T, Mar S, Valeriani M, et al. Ocrelizumab dose selection for treatment of pediatric relapsing-remitting multiple sclerosis: pharmacokinetic, safety, and efficacy results from OPERETTA 1. Presented at: 2024 CMSC Annual Meeting; May 29-June 2, 2024; Nashville, TN. Abstract DMT02. https://cmscscholar.org
4. Hauser SL, Bar-Or A, Comi G, et al. Ocrelizumab versus interferon beta-1a in relapsing multiple sclerosis. N Engl J Med. 2017;376(3):221-234. doi:10.1056/NEJMoa1601277. https://www.nejm.org/doi/full/10.1056/NEJMoa1601277
5. Genentech. FDA approves Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for relapsing and progressive multiple sclerosis. News release. September 2024. Accessed May 2026. https://www.gene.com/media/press-releases